Key clinical point: A single infusion of SPK-8011 reduced bleed and infusion rates in severe hemophilia A patients.
Major finding: The overall reduction in annualized infusion rate (AIR) and annualized bleeding rate (ABR) was 97% for each.
Study details: A phase 1/2 study of 12 patients
Disclosures: Spark Therapeutics sponsored the study. Dr. George reported equity ownership related to the University of Pennsylvania and consultancy for Pfizer.
High K et al. ASH 2018, Abstract 487.